New hope for rare bleeding disorder: BIVV001 tested in humans
NCT ID NCT04770935
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tested a single injection of a new drug called BIVV001 in 6 adults with severe forms of von Willebrand disease (types 2N and 3). The goal was to see how the drug behaves in the body and whether it is safe. If results are promising, larger studies could follow to see if it helps control bleeding.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- efanesoctocog alfa (BIVV001)
- What this could lead to
- If successful, this could lead to a new treatment option that helps control bleeding episodes in people with severe von Willebrand disease.
- What could go wrong
- This is a very early, small Phase 1 trial with only 6 participants, so results may not apply to everyone. The drug may not work as hoped or could cause unexpected side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Hemophilia Center of Western Pennsylvania_Investigational Site Number :8400001
Pittsburgh, Pennsylvania, 15213, United States
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Investigational Site Number :2500001
Lille, 59037, France
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Investigational Site Number :2500002
Nantes, 44093, France
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University of Iowa_Investigational Site Number :8400002
Iowa City, Iowa, 52242, United States